Exploring Gene Therapy Approaches and Treatments for Various Diseases

Dr. David Williams, Chief of the Division of Hematology & Oncology at Boston Children’s Hospital, Associate Chairman of the Department of Pediatric Oncology at the Dana-Farber Cancer Institute, and Leland Fikes Professor of Pediatrics at Harvard Medical School

March 15, 2024

In this episode, we discuss advances in gene therapy and its use in treating pediatric patients. Using modified viruses as delivery vehicles, researchers and clinicians can target therapies to specific types of cells, providing patients with long-lasting—and potentially life-long—treatments for a variety of severe genetic conditions. Future advances hope to make these therapies less expensive, less invasive, and more accessible for patients around the world and to expand their use for more common conditions.